Longevity Leaders Conference. February 4, 2019

Transcription

1 Longevity Leaders Conference February 4, 2019

2 Forward Looking Statements The matters discussed in this presentation include forward looking statements which are subject to various risks, uncertainties, and other factors that could cause actual results to differ materially from the results anticipated. Such risks and uncertainties include but are not limited to the success of AgeX Therapeutics and its affiliates in developing new stem cell-based products and technologies; results of clinical trials of such products; the ability of AgeX and its licensees to obtain additional FDA and foreign regulatory approval to market products; competition from products manufactured and sold or being developed by other companies; the price of and demand for such products; the ability of AgeX and its subsidiaries to maintain patent and other intellectual property rights; and the ability of AgeX to raise the capital needed to finance its current and planned operations. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. As actual results may differ materially from the results anticipated in these forward-looking statements they should be evaluated together with the many uncertainties that affect the business of AgeX and its other subsidiaries, particularly those mentioned in the cautionary statements found in AgeX's Securities and Exchange Commission filings. AgeX disclaims any intent or obligation to update these forward-looking statements. 2 2

3 Mission AgeX Therapeutics is focused on the development of young cell-based regenerative therapies for the treatment of human aging. 3

4 The Target Market Aging and chronic degenerative disease 80% of $2.5T health care costs associated with chronic disease. 80% of elderly have at least one chronic disease, 68% have two or more. 4

5 5 The Ideal Technology Platform Young replacement cells of all kinds Cells capable of regeneration A path to an off-the-shelf product An injectable mix of cells/matrix to regenerate 3-D tissue + = Regenerative Medicine For Age-Related Degenerative Disease Young Cells Matrix 5

6 The Ideal Technology Platform Twin Technologies: Cells & Matrix Pluripotent Cell- Based Therapeutics U.S. projected >80 yrs. old HyStem Matrix Delivery Pluripotent Stem Cells (PSCs) allow the manufacture of all young human cell types on an industrial scale Engineered for allogeneic use Our cells are government (NIH) approved Biomaterials Jul; 34(22):

7 7 History of the Biotechnology Revolutions Recombinant DNA Technology Monoclonal Antibodies Regenerative Medicine 1974 Gene cloning technology developed Moratorium on rdna research initiated led to established guidelines on rdna research 1989 First $B product EPO Today, products from the use of rdna technology are ubiquitous >140 clinical trials Current Global Market $75 B Hybridoma technology developed First $B Product Rituximab Advances in Mab Engineering Today, eight of the 20 bestselling biotechnology drugs in therapeutic monoclonal antibodies > 200 clinical trials Current Global Market $44 B 1998 First Pluripotent Stem Cells isolated 2001 U.S. Federal funding restriction (reversed in 2009) st hes Clinical trial Future 1 st $B product 7

8 Numerous Products Performing Well in Trials Retinal Pigment Epithelial cells (OpRegen) Agerelated macular degeneration (BioTime Phase II) Oligodendrocyte Progenitor Cells (OPC1) Spinal cord injury (Asterias Phase II) Dendritic cells (VAC2) Cancer immunotherapy (Asterias/CRUK Phase I) 8

9 Pluripotency The Competitive Edge The >1000-fold complexity of cell types derived from hps cells leads to unique challenges: How manufacture with cgmp? How produce allogeneic product? Identity - Lot-to-lot variability in composition Purity Contamination with unknown cell types Desired Cell Type Embryoid Body Activin A FGF2 TGFb3 9

10 cgmp Manufacture U.S. projected >80 yrs. old First publication describing the derivation of clinical-grade GMP hes cell lines Comprehensive, multiple stage donor consent FDA approved, GMP human fibroblast feeder cell line Six karyotypically normal hes cell lines successfully derived Screened for panel of adventitious agents NIH Registered 10

11 UniverCyte TM : HLA-G for Allogeneic Immunotolerance It appears that the primary role of HLA-G is to suppress maternal immune response to pregnancies. Appears to disarm multiple arms of immune system Adv. Immunol. (2015) 127:

12 Value of the UniverCyte Pluripotent Platform Classical biologics off-the-shelf business model Traditional Biologicals Centralized Production Facility Distributed Frozen Inventory Point Of Care UniverCyte- Derived Cell Therapy Products 12

13 PureStem Technology Traditional Manufacture Universal PureStem TM Technology ES Cells Differentiation Purification of desired cell type Problem of impurities 13

14 Identification of Low-Hanging Fruit Key applications in age-related degenerative disease Disease characterized by loss of cells Not addressable with current modalities U.S. projected >80 yrs. old 14

15 Brown Adipose Cells Regulate Metabolism WAT Weight Gain T2 Diabetes Maintenance Weight Loss BAT 15

16 Brown Adipose Cells Regulate Metabolism Obesity (2011) 19, doi: /oby

17 AgeX-BAT1 Properties Stained for Brown Adipocyte Marker UCP1 Tissue-Sourced Brown Adipocytes PureStem Brown Adipocytes West et al. Stem Cell Research & Therapy (2019) 10:7 17

18 Obesity/T2D Market/Competition U.S. projected >80 yrs. old 30M Americans have diabetes 1 1:3 Americans will have diabetes by 2050 The global market for diabetes mellitus and obesity is set to rise from $70.8 billion in 2015 to $163.2 billion by 2022, at a strong compound annual growth rate of 12.7%, according to business intelligence firm GBI Research. Competing products commonly target insulin secretion, glucose excretion, incretins such as GLP-1, or attempt to activate existing BAT or cause browning of white fat. 1) Centers for Disease Control and Prevention. National Diabetes Statistics Report: Estimates of Diabetes and Its Burden in the United States. US Department of Health and Human Services; Atlanta, GA:

19 AgeX-VASC1 Purity Monoclonal Endothelium ~100% Purity GFP Endothelium (168 hrs) 19

20 Cardiovascular Market > $Trillion Market Worldwide U.S. projected >80 yrs. old 20

21 Induced Tissue Regeneration (itr) Innate regeneration in humans restricted to early development 21

22 Reversing the Aging of Human Cells Back to Pluripotency Reprogramming Methylation Age Skin Fibroblasts ips Cells Regen Med 2010 May;5(3): Genome Biol. 2013;14(10):R115 22

23 Reversing the Aging of Human Cells Back to Regeneration Repair > Breakdown Repair = Breakdown Breakdown > Repair Development Adult Aging Adult Highly Regenerative Limited Regeneration Non-Regenerative Construction Maintenance itr: induced Tissue Regeneration Destruction 23

24 An Example of an itr Gene Pluripotent (off) AGEX-iTR1547 Embryonic/ Fetal Levels 24

25 Product Pipeline THERAPEUTICS AGEX-BAT1 (Brown Adipocytes) Pre-Clinical Phase I Phase II Phase III/Pivotal U.S. projected >80 yrs. old T2D AGEX-VASC1 (Vascular Progenitors) AGEX-iTR1547 (NCE in HyStem) Renelon TM (Repurposed Drug) 510(k) MI CHF Scarless Healing 510(k) Clearance RESEARCH PRODUCTS Universal cgmp ES Cells, Cytiva DATABASE PRODUCTS GeneCards/LM Discovery CANCER DIAGNOSTICS & THERAPY Cancer Stem Cell EFT Dx & Tx Marketed Research Products Marketed NGS Interpretation To be Partnered for Cancer Dx 25

26 Summary Largest challenge is chronic degenerative diseases of aging Straightforward therapeutic strategy: Young cells for aged tissues Proprietary manufacturing technology yielding highly scalable, purified, identified, and regenerative cells for applications in age-related degenerative disease A proprietary path to off-the-shelf allogeneic application A proprietary injectable matrix itr has potential to induce not only regeneration in aged tissues but also induce senolysis 26